SAN DIEGO, Feb. 07, 2018 (GLOBE NEWSWIRE) -- Cures for sickle cell, hemophilia and a host of other genetic-based diseases are on the horizon because of nanotechnology that can now deliver CRISPR, mRNA, and other genetic tools to precise locations inside the cell.

“Our technology is a fundamental breakthrough,” Andre Watson, founder and CEO of Ligandal told health and science leaders at the Cavendish Health Impact Forum in San Diego today. “For the first time, we can get CRISPR and other genetic therapies to where they need to go. What good would Amazon be without the shipping?”

Ligandal uses the power of ligands – molecules that bind with specific other molecules – as well as a breakthrough nanomedicine platform to target the precise molecular ‘zip codes’ (receptors) of cells and their nuclei.

“Most existing drugs either utilize viruses or recombinant protein factors,” Watson said. “Viral gene therapies and other current techniques pose significant concerns with immunogenicity and other safety issues. On the other hand, we’ve developed nanoparticles that have no inherent immunogenicity or toxicity, enable precise engineering and flexible payloads, and can time-release the delivery of new characteristics into the correct organelle of the cell.”

Watson said that ligands combined with Ligandal’s next-generation, non-viral, protein-based biomaterials will soon deliver therapies enabled by CRIPSR, TALEN, mRNA, siRNA, DNA, and the future of genetic technologies.

The need for such a delivery system is hard to overestimate.

  • Eight percent of the U.S. population has a genetic disease caused by a single mutation. Other diseases, though not specifically genetic, have molecular causes. About 400 therapies exist for nearly 8,000 rare diseases, most of which are caused by a single gene.
  • One-sixth of genetic diseases have therapies, and many of those have serious complications. Orphan drugs to treat these diseases are the most expensive pharmaceuticals in the world, costing up to $700,000 a year.

Pre-clinical studies of Ligandal technology indicate that it will create unprecedented freedom to innovate in sickle cell disease, β-thalassemia, osteogenesis imperfecta, hemophilia, as well as with cancer immunotherapy, Watson said.

The market potential for this technology is also hard to overestimate.

  • Rare blood diseases are projected to represent a $16 billion market by 2022.
  • Hematological malignancies and solid tumors are projected to represent a $169 billion market by 2025.
  • Treating the 200,000 sickle-cell patients in the United States throughout their lives is estimated to cost $1 trillion.

“Beyond monogenetic diseases, we foresee our technology opening a frontier of genetically tailored, molecularly precise medicine,” Watson said. “Our technology also creates the infrastructure for rapid gene therapy discovery and drug development.”

Therapies based on nanoparticles will be easily customized, rapidly scalable, and provide the potential to bring down production costs by 100 times, he said.

Watson pointed to several datasets that show proof of concept, including the deletion of a mouse gene in vivo, the insertion of a gene into mouse bone marrow ex vivo, efficient delivery of CRISPR and mRNA into ex vivo cynomolgus bone marrow, as well as 99% efficient delivery of CRISPR to human T-cells.

Ligandal is in the process of completing datasets surrounding numerous indications in hematology and immuno-oncology, which will give the company unprecedented latitude in the gene therapy landscape.

ABOUT LIGANDAL
Based in San Francisco, California, Ligandal is a nanomedicine company developing unique curative genetic therapies with a personalized nanotechnology platform. Visit www.ligandal.com to learn more.